Alagille Syndrome
FDA expands Livmarli approval for pruritus in PFIC to infants as young as 12 months
The FDA has updated the label indication for Livmarli for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis, expanding the patient population to include infants aged 12 months and older.
ASSERT: Bylvay outperforms placebo in reducing pruritis, bile acids in Alagille syndrome
Treatment with Bylvay resulted in a “significantly greater” change from baseline to week 24 in mean scratching scores and serum bile acids vs. placebo among pediatric patients with Alagille syndrome, according to ASSERT study results.
FDA approves Livmarli for pruritus in progressive familial intrahepatic cholestasis
The FDA has approved Mirum Pharmaceuticals’ Livmarli oral solution for treatment of cholestatic pruritus in patients aged 5 years and older with progressive familial intrahepatic cholestasis, according to a company release.
‘Show your stripes:’ Rare Disease Day allows patients to share their stories, spread awareness
More than 300 million people worldwide, including one in every 10 Americans, are living with a rare disease, yet these individuals and their families are often left isolated and without answers.
VIDEO: Livmarli reduces serum bile acids and pruritus in PFIC, Alagille syndrome
In a Healio video exclusive, Pam Vig, PhD, highlights new data on Livmarli for the treatment of patients with pruritus due to progressive familial intrahepatic cholestasis and Alagille syndrome, which were presented at EASL Congress.
FDA approves Bylvay for cholestatic pruritus due to Alagille syndrome
The FDA has approved Bylvay for the treatment of cholestatic pruritus due to Alagille syndrome in patients aged 12 months or older, according to an Ipsen Biopharmaceuticals press release.
FDA expands Livmarli label to include infants with pruritus aged 3 months or older
The FDA has approved labeling changes for Livmarli, reducing the age of prescribing to infants with Alagille syndrome from 1 year to 3 months for the treatment of cholestatic pruritus, according to a Mirum Pharmaceuticals press release.
European Commission approves Livmarli for patients with Alagille syndrome
Mirum Pharmaceuticals announced the European Commission has approved Livmarli for the treatment of cholestatic pruritis in patients with Alagille syndrome.
VIDEO: Bylvay improves pruritus, reduces serum bile acids in Alagille syndrome
WASHINGTON D.C. — In a Healio video exclusive, Ron Cooper, CEO of Albireo Pharma, highlighted positive top-line safety and efficacy results of Bylvay for the treatment of Alagille syndrome in patients from birth to early adulthood.